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Clinical Trials in Ireland / NCT05883748
Enrolling by invitation Phase 2/3

HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP

NCT05883748 · tracked via the Priya Life Science Ireland tracker
Sponsor
Disc Medicine, Inc
Phase
Phase 2/3
Started
2023-08-31
Last updated
2026-09-10

Condition(s) studied

Erythropoietic Protoporphyria

Investigational drug(s) / intervention(s)

DISC-1459DISC-1459

DISC-1459: DISC-1459 dose level 1

DISC-1459: DISC-1459 dose level 2

Study summary

This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

Eligibility

Sex
ALL
Min age
12 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit * Aged ≥12 years upon study consent * Body weight ≥32 kg for participants \<18 years of age and BMI ≥18.5 kg/m2 for adult participants * Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug. * Negative urine or serum pregnancy test (females of childbearing potential). * Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary). * Able to comply with all study procedures. Exclusion Criteria: * Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin * Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study * Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months * Planned treatment with afamelanotide or dersimelagon during the study * Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study * If female, pregnant, or breastfeeding * Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1 * Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years. * Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study

Primary outcome measure(s)

Trial sites (26)

FacilityCityRegionStatus
University of Alabama Hospital Birmingham Alabama
University of California San Francisco San Francisco California
University of Miami Miller School of Medicine Miami Florida
Massachusetts General Hospital Boston Massachusetts
MetroBoston Clinical Partners Boston Massachusetts
Mount Sinai Hospital New York New York
Atrium Health Wake Forest Baptist Winston-Salem North Carolina
Remington-Davis Clinical Research Columbus Ohio
Einstein Medical Center Philadelphia Pennsylvania
University of Texas Galveston Texas
Fred Hutchinson Cancer Center Seattle Washington
Royal Prince Alfred Hospital Camperdown New South Wales
The Royal Melbourne Hospital Parkville Victoria
UZ Leuven Leuven Belgium
CHU de Nantes - Hôtel Dieu, Service de dermatologie Nantes France
Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard Paris France
Charité - Universitätsmedizin Berlin, Institute of Allergology Berlin Germany
Klinikum Chemnitz gGmbH Chemnitz Saxony
Children's Health Ireland (CHI) Dublin Ireland
Instituto Dermatologico San Gallicano Istituti Fisioterapici Ospitalieri IRCCS Roma Italy
Erasmus MC Rotterdam Netherlands
Hospital Clinic de Barcelona Barcelona Spain
Karolinska University Hospital Stockholm Sweden
Guy's and St Thomas' NHS Foundation Trust London England
Clinical Research Centre, Ninewells Hospital & Medical School , NHS Tayside Dundee Scotland
Photobiology Unit, Salford Royal Hospital Salford United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05883748 on ClinicalTrials.gov ↗ ← All trials in Ireland